| Allogeneic hematopoietic stem cell transplantation from an alternative stem cell source in Fanconi anemia patients: analysis of 47 patients from a single institution. | |
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MedLine Citation:
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PMID: 17053839 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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We transplanted 47 patients with Fanconi anemia using an alternative source of hematopoietic cells. The patients were assigned to the following groups: group 1, unrelated bone marrow (N = 15); group 2, unrelated cord blood (N = 17), and group 3, related non-sibling bone marrow (N = 15). Twenty-four patients (51%) had complete engraftment, which was not influenced by gender (P = 0.87), age (P = 0.45), dose of cyclophosphamide (P = 0.80), nucleated cell dose infused (P = 0.60), or use of anti-T serotherapy (P = 0.20). Favorable factors for superior engraftment were full HLA compatibility (independent of the source of cells; P = 0.007) and use of a fludarabine-based conditioning regimen (P = 0.046). Unfavorable factors were > or = 25 transfusions pre-transplant (P = 0.011) and degree of HLA disparity (P = 0.007). Intensity of mucositis (P = 0.50) and use of androgen prior to transplant had no influence on survival (P = 0.80). Acute graft-versus-host disease (GVHD) grade II-IV and chronic GVHD were diagnosed in 47 and 23% of available patients, respectively, and infections prevailed as the main cause of death, associated or not with GVHD. Eighteen patients are alive, the Kaplan-Meyer overall survival is 38% at approximately 8 years, and the best results were obtained with related non-sibling bone marrow patients. Three recommendations emerged from the present study: fludarabine as part of conditioning, transplant in patients with < 25 transfusions and avoidance of HLA disparity. In addition, an extended family search (even when consanguinity is not present) seeking for a related non-sibling donor is highly recommended. |
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Authors:
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C R de Medeiros; M A Bitencourt; J Zanis-Neto; E C P Maluf; D S Carvalho; C S Bonfim; V M Funke; D C Setubal; N Farah; R Pasquini |
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Publication Detail:
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Type: Journal Article |
Journal Detail:
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Title: Brazilian journal of medical and biological research = Revista brasileira de pesquisas médicas e biológicas / Sociedade Brasileira de Biofísica ... [et al.] Volume: 39 ISSN: 0100-879X ISO Abbreviation: Braz. J. Med. Biol. Res. Publication Date: 2006 Oct |
Date Detail:
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Created Date: 2006-10-20 Completed Date: 2007-08-02 Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 8112917 Medline TA: Braz J Med Biol Res Country: Brazil |
Other Details:
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Languages: eng Pagination: 1297-304 Citation Subset: IM |
Affiliation:
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Serviço de Transplante de Medula Ossea, Hospital de Clínicas, Universidade Federal do Paraná, Rua General Carneiro 181, 80060-900 Curitiba, PR, Brazil. crdemedeiros@terra.com.br |
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| MeSH Terms | |
Descriptor/Qualifier:
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Acute Disease Adolescent Adult Child Child, Preschool Chronic Disease Cyclophosphamide / therapeutic use Fanconi Anemia / therapy* Female Graft vs Host Disease / diagnosis, prevention & control HLA Antigens / analysis Hematopoietic Stem Cell Transplantation / methods* Histocompatibility Testing Humans Immunosuppressive Agents / therapeutic use Male Multivariate Analysis Retrospective Studies Severity of Illness Index Time Factors Transplantation Conditioning / methods* Transplantation, Homologous / immunology, methods Treatment Outcome |
| Chemical | |
Reg. No./Substance:
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0/HLA Antigens; 0/Immunosuppressive Agents; 50-18-0/Cyclophosphamide |
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